Orchard Therapeutics Advances New Strategic Plan and Reports Second Quarter 2020 Financial Results
Regulatory and Clinical Progress in MLD, MPS-I and MPS-IIIA Illustrate Execution in Neurometabolic Disorders; EU Regulatory Decision for Libmeldy™ Expected in 2H 2020
MolMed and GSK Agreements Provide Near-term Manufacturing Capacity and Highlight Future Innovations
Near-term Financial Position Secure with Cash and Investments of
“Implementing Orchard’s new strategic plan yielded strong results in the second quarter as we marked important regulatory and clinical progress in our neurometabolic programs and strengthened our current and future manufacturing position,” said
Recent Corporate Achievements Supporting New Strategic Plan
Prioritize portfolio investments to realize opportunities for high need, high value indications, with an emphasis on neurometabolic conditions
- Libmeldy (OTL-200) for MLD:
- Submitted responses to the Day 120 questions from the
European Medicines Agency (EMA) for the marketing authorization application (MAA) for Libmeldy (OTL-200) for the treatment of metachromatic leukodystrophy (MLD).
- Submitted responses to the Day 120 questions from the
- OTL-203 for MPS-I:
- Received orphan drug and rare pediatric disease designations for the treatment of mucopolysaccharidosis type I (MPS-I) from the
U.S. Food and Drug Administration (FDA). - Presented interim data from the ongoing proof-of-concept trial of OTL-203 at the
American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting. The first primary outcome measure of the study was met with all eight patients achieving hematologic engraftment within 45 days of treatment. In addition, clinical data from the first two patients treated have shown improved motor function, stable cognitive scores and continued normal growth after at least one year of follow-up. Link to full release here.
- Received orphan drug and rare pediatric disease designations for the treatment of mucopolysaccharidosis type I (MPS-I) from the
- OTL-201 for MPS-IIIA:
- Enrolled three patients in the proof-of-concept study of OTL-201 for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).
Establish focused commercial model for diagnosis and treatment of patients globally
- Extended the gene therapy manufacturing relationship with MolMed S.p.A (an AGC Inc. company) for a period of five years through
June 2025 . MolMed will continue to support activities related to the development and manufacturing of vectors and drug product for multiple Orchard investigational programs, including for the potential upcoming commercial launch of Libmeldy (OTL-200) for MLD. Link to full release here.
Invest in next-generation manufacturing technology and process innovations
- Entered into worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) for use of their proprietary lentiviral stable cell line technology in gene therapy products for the treatment of Wiskott Aldrich syndrome (WAS) and transfusion-dependent beta thalassemia (TDT). Link to full release here.
Planned Corporate Milestones
A Media Snippet accompanying this announcement is available by clicking on the image or link below:
Second Quarter 2020 Financial Results
Research and development expenses were
Selling, general and administrative expenses were
Net loss was
Cash, cash equivalents and investments as of
Conference Call & Webcast Information
Orchard will host a conference call and live webcast today at
About Orchard
Orchard has its global headquarters in
Availability of Other Information About Orchard
Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets,
Forward-Looking Statements
This press release contains certain forward-looking statements about Orchard’s strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as “anticipates,” “believes,” “expects,” “plans,” “intends,” “projects,” and “future” or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchard’s business strategy and goals, the therapeutic potential of Orchard’s product candidates, including the product candidates referred to in this release, Orchard’s expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, the likelihood of approval of such product candidates by the applicable regulatory authorities, Orchard’s expectations concerning its partnership with MolMed, Orchard’s expectations regarding its expected use of stable cell line technology and the potential benefits of its agreements with GSK, and Orchard’s financial condition and cash runway into 2022. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchard’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the severity of the impact of the COVID-19 pandemic on Orchard’s business, including on clinical development, its supply chain and commercial programs; the risk that Orchard will not realize the anticipated benefits of its new strategic plan or the expected cash savings associated with such plan; the risk that any one or more of Orchard’s product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk of cessation or delay of any of Orchard’s ongoing or planned clinical trials; the risk that Orchard may not successfully recruit or enroll a sufficient number of patients for its clinical trials; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchard’s product candidates or that long-term adverse safety findings may be discovered; the delay of any of Orchard’s regulatory submissions; the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchard’s product candidates or the receipt of restricted marketing approvals; the risk of delays in Orchard’s ability to commercialize its product candidates, if approved; and the risk that the market opportunity for its product candidates may be lower than estimated. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchard’s quarterly report on Form 10-Q for the quarter ended
Condensed Consolidated Statements of Operations
(In thousands, except share and per share amounts)
(unaudited)
| Three Months Ended |
Six Months Ended |
|||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Product sales, net | $ | 597 | $ | — | $ | 597 | $ | — | ||||||||
| Costs and operating expenses: | ||||||||||||||||
| Cost of product sales | 191 | — | 191 | — | ||||||||||||
| Research and development | 31,568 | 40,478 | $ | 56,404 | 57,971 | |||||||||||
| Selling, general and administrative | 15,659 | 13,674 | 35,804 | 24,464 | ||||||||||||
| Total costs and operating expenses | 47,418 | 54,152 | 92,399 | 82,435 | ||||||||||||
| Loss from operations | (46,821 | ) | (54,152 | ) | (91,802 | ) | (82,435 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 892 | 1,727 | 2,372 | 3,350 | ||||||||||||
| Interest expense | (568 | ) | (245 | ) | (1,181 | ) | (245 | ) | ||||||||
| Other income (expense), net | (943 | ) | 1,368 | (7,733 | ) | (2,118 | ) | |||||||||
| Total other income (expense), net | (619 | ) | 2,850 | (6,542 | ) | 987 | ||||||||||
| Net loss before income tax | (47,440 | ) | (51,302 | ) | (98,344 | ) | (81,448 | ) | ||||||||
| Income tax (expense) benefit | (60 | ) | 772 | 275 | 179 | |||||||||||
| Net loss attributable to ordinary shareholders | (47,500 | ) | (50,530 | ) | (98,069 | ) | (81,269 | ) | ||||||||
| Net loss per share attributable to ordinary shareholders, basic and diluted | $ | (0.48 | ) | $ | (0.56 | ) | $ | (0.99 | ) | $ | (0.92 | ) | ||||
| Weighted average number of ordinary shares outstanding, basic and diluted | 99,251,314 | 89,712,916 | 99,048,498 | 88,369,311 | ||||||||||||
Condensed Consolidated Balance Sheet Data
(In thousands)
(unaudited)
| 2020 | 2019 | |||||
| Assets | ||||||
| Cash, cash equivalents and marketable securities | $ | 228,654 | $ | 324,990 | ||
| Trade receivables | 723 | 1,442 | ||||
| Research and development tax credit receivable | 32,729 | 28,644 | ||||
| Prepaid expenses and other current assets | 12,760 | 8,530 | ||||
| Operating lease right-of-use assets | 18,642 | 19,415 | ||||
| Property and equipment, net | 5,460 | 7,596 | ||||
| Other assets | 15,608 | 8,664 | ||||
| Total assets | $ | 314,576 | $ | 399,281 | ||
| Liabilities and shareholders' equity | ||||||
| Accounts payable | $ | 15,590 | $ | 11,984 | ||
| Accrued expenses and other current liabilities | 19,811 | 37,980 | ||||
| Operating lease liabilities | 23,095 | 21,212 | ||||
| Long-term debt, net | 24,886 | 24,699 | ||||
| Other long-term liabilities | 4,173 | 4,213 | ||||
| Total liabilities | 87,555 | 100,088 | ||||
| Shareholders’ equity | 227,021 | 299,193 | ||||
| Total liabilities and shareholders’ equity | $ | 314,576 | $ | 399,281 | ||
Contacts
Investors
Director, Investor Relations
+1 862-242-0764
Renee.Leck@orchard-tx.com
Media
VP, Corporate Affairs
+1 202-415-0137
media@orchard-tx.com
Source: Orchard Therapeutics (Europe) Limited